December 2, 2011 — Lysosomal storage disorders such as Fabry's disease and Pompe's disease are much more common than previously thought, particularly atypical later-onset forms, a new study suggests.

S03, a cell-based gene therapy designed to replace 130 lifetime Fabry infusions with a single procedure. FDA’s acceptance of INTERACT request validates the clinical and manufacturing case built on ...

Rare diseases often remain undetected for years, mostly because of limited awareness and complex symptoms that are not easy to diagnose. One such group of conditions is Lysosomal Storage Disorders, or ...

Main takeaways: Scientists at the Icahn School of Medicine at Mount Sinai in New York, the National Center for Advancing Translational Sciences (NCATS), and elsewhere have reversed the effects of ...

Please provide your email address to receive an email when new articles are posted on . An Italian pharmaceutical company and a Boston-area biotechnology research firm announced a partnership to ...

Cholesteryl Ester Storage Disease (CESD) is a rare autosomal recessive multisystem condition that belongs to the lysosomal storage disorders family. The liver is one of the most common sites of ...

Azafaros, a private company building a portfolio to become a leader in Lysosomal Storage Disorders and focused on addressing neurological symptoms, today announced that Amy Sullivan has joined the ...

Artificial intelligence is becoming increasingly important in drug discovery. Advances in the use of Big Data, learning algorithms and powerful computers have now enabled researchers at the University ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE: 4552) made significant contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024, held ...